Oral: Benefit-Risk assessment

B-05 Current practices and gaps in benefit-risk assessment: opportunities for combining MCDA with model-based approaches

Kevin Marsh

Evidera

Objectives:

• Provide an overview of the use of quantitative BRA by the FDA.
• Illustrate how quantitative BRA is undertaken by industry to demonstrate the value of technologies.
• Illustrate how quantitative BRA can be incorporated into model-based approaches to trial design. 
• Identify the challenges, and potential solutions to using quantitative BRA to inform trial design.

Overview/Description of presentation: Industry’s use of quantitative benefit-risk assessment (BRA) has recently received stimulus from an increased interest in its use by the FDA. Most BRA is thus undertaken by industry alongside phase 3 trials. However, there is increasing interest in undertaking BRA earlier in the development process, incorporating BRA into model-based approaches to informing the design of trials. This will allow the assessment of trial design scenarios to be based on models of the probability of achieving regulatory approval.

The presentation will be organised into four sections:

1. A definition of quantitative BRA, how this differs from other types of BRA, and how MCDA supports quantitative BRA.
2. A summary of FDA requirements and guidelines for quantitative BRA, and an illustration of a BRA used in an FDA approval decision.
3. An outline of how quantitative BRA could be incorporated into model-based approaches to trial design.
4. An outline of key considerations when designing a quantitative BRA for incorporation into model-based approaches to trial design, including dealing with uncertainty in performance ranges, and generating weights for longer lists of treatment attributes.

Conclusions/Take home message: Quantitative BRA is increasingly being used to support approval in the US. Anticipating this requirement, it is possible to incorporate quantitative BRA into model-based approaches to trial design.